A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Part of paid clinical trials in Oakland, California.

Sponsor
Aspa Therapeutics
Study ID
NCT04998396
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Canavan Disease

Eligibility Criteria

Sex
ALL
Age
N/A - 30 Months
Healthy Volunteers
Not accepted

Interventions

  • AAV9 BBP-812 — BIOLOGICAL
    Sterile solution for injection for 1-time use via volumetric infusion pump

Study Details

The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

Key Dates

Start date
Sep 8, 2021
Status verified
Apr 2026
Primary completion
Oct 13, 2026
Completion
Oct 8, 2032

Study Design

Enrollment
26 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)
    Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
  • Experimental: Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)
    Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
  • Experimental: Enrollment Expansion Phase: BBP-812
    Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.

Primary Outcome Measure

Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Week 52 ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609
Marissa Evans
[email protected]
510-428-3885
Grace Sobrero
[email protected]
510-428-3885
Alexander Fay, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Prachi Patel
[email protected]
312-227-0067
Jennifer Rubin, MD (PRINCIPAL_INVESTIGATOR)
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)BostonMassachusetts02114
Sam Kartsonis
[email protected]
610-766-2583
Aliza (Hattie) Wilson
[email protected]
Florian Eichler, MD (PRINCIPAL_INVESTIGATOR)
Weill Cornell Medicine; Division of Pediatric NeurologyNew YorkNew York10065-

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILMassachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)· Boston, MAWeill Cornell Medicine; Division of Pediatric Neurology· New York, NY

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